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Roche Buys Spinal Muscular Atrophy Pipeline
November 29, 2011
Swiss pharmaceutical giant Roche makes a large investment in the neurodegenerative disease space with its $490 million acquisition of exclusive rights to NJ-based PTC Therapeutics' program for spinal muscular atrophy (SMA). The arrangement gives Roche global rights to three preclinical compounds in PTC’s SMA pipeline, which were developed in collaboration with the SMA Foundation.


Amorfix to Develop a Blood Test for ALS
November 17, 2011
Amorfix Life Sciences, a Canadian biotech company focused on diagnostics and therapeutics for misfolded protein diseases, announced that it is developing a diagnostic blood test for ALS based on the finding that misfolded SOD1 is present in the blood of ALS patients. The company plans to capitalize on this discovery and existing scientific expertise to develop a simple blood test that measures misfolded SOD1 in blood. Amorfix is also partnering with Biogen Idec on a potential ALS therapy.


Pfizer's Drug for Rare Neuropathology Get Approved in EU, Not US
November 17, 2011
Pfizer's Vyndaqel (tafamidis) was approved by the European Commission for the treatment of transthyretin familial amyloid polyneuropathy (TTR-FAP), a progressive and fatal neurodegenerative disease affecting approximately 8,000 patients worldwide. Vyndagel was shown to be efficacious in delaying peripheral neurologic impairment as well as alleviating the digestive problems associated with the disease. It is expected to be launched in Europe by early 2012 but approval in the US will require additional data requested by the FDA in April.


Geron Is Shutting Down Its Stem Cell Clinical Trial
November 14, 2011
California based biotech Geron, which had been conducting the world's first clinical trial of a human embryonic stem cell-based therapy for spinal cord injury, announced it was halting its trial and leaving the stem cell field all together. Geron attributed financial difficulties rather than any problems with the trial itself to its exit from the stem cell therapeutics areana. However, the move is expected to be widely seen as a setback for the field, already facing controversy around the use of human embryonic tissue. Advanced Cell Technology, also facing financial difficulties, is now the only company conducting a clinical trial involving human embryonic stem cells.


ReNeuron's Stem Cell Treatment for Stroke Advances
November 9, 2011
Scottish biotech ReNeuron announced that the first ever stroke patient to be treated with its stem cell therapy remains well a year later with no "cell-related adverse events reported" after 28 days of treatment. The success enables ReNeuron to advance to a second cohort of patients, who will receive higher doses. In all, 12 patients will be given varying doses of ReNeuron's ReN001 stem cell treatment as part of the Scottish phase I trial. ReNeuron derives its stem cell products from non-embryonic human tissue sources.